An Investigator-initiated study on rFVIIa prophylaxis in children with haemophilia A and inhibitors

Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico0 个研究点目标入组 50 人开始时间: 2012年7月26日最近更新: 2017年10月2日

适应症

试验速览

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•Male patients with haemophilia A who have been treated with FVIII on demand or on prophylaxis and who have developed inhibitors to FVIII.
•Age < 8 years.
•= 2 years from the time of first inhibitor detection.
•High-responding inhibitors (historical peak > 5 BU/mL) and known anamnestic response in case of negative inhibitor titre.
•Patient is candidate for daily ITI treatment with doses of FVIII ranging from 50 IU/kg/day to 200 IU/kg/day.
•Maximal two bleedings in the same joint within the last 6 months before entering the study or maximal six joint bleeds in the same joint within 2 years.
•Adequate venous access for daily infusion and capable (patient or caregiver) of reconstituting and injecting the study drug (as judged by the Investigator).
•Informed consent by parents or legal guardians.
•Are the trial subjects under 18? yes
•Number of subjects for this age range:
•F.1.2 Adults (18-64 years) no
•F.1.2.1 Number of subjects for this age range
•F.1.3 Elderly (>=65 years) no
•F.1.3.1 Number of subjects for this age range

•ITI already started.
•Known or suspected hypersensitivity to the active substance, or to any of the excipients of the study drug, or to mouse, hamster or bovine protein.
•Administration of any investigational product within 30 days prior to randomisation.
•Other coagulation disorders than congenital haemophilia A.
•Family history of thrombosis at an early age (< 40 years), known thrombophilia, any previous thrombosis including catheter-related deep vein thrombosis, previous neonatal thrombosis.
•Known pseudo tumours.
•Known severe liver disease.
•Platelet count < 50,000 platelets/µL at screening.
•Surgery within one month or planned major and/or orthopaedic surgery.

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